Date: 2015-10-01
Type of information: Licensing agreement
Compound: Kuvan®, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’, pegvaliase)
Company: Merck KGaA (Germany) Biomarin Pharmaceutical (USA - CA)
Therapeutic area: Rare diseases - Genetic diseases - Metabolic diseases
Type agreement: licensing development commercialisation
Action mechanism: enzyme co-factor. Kuvan® is the synthetic form of 6R-BH4, a naturally occurring co-factor that works in conjunction with the enzyme phenylalanine hydroxylase (PAH) to metabolize phenylalanine (Phe) into tyrosine. Clinical data show that Kuvan® produces significant reductions in blood Phe concentration in a large subset of patients. enzyme replacement therapy. PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase or ‘PAL’, pegvaliase) is an investigational drug that substitutes for the PAH enzyme in phenylketonuria (PKU). PEG-PAL is being developed as a potential treatment for patients whose blood phenylalanine levels are not adequately controlled by Kuvan® or who have trouble controlling and maintaining their phenylalanine levels).
European marketing authorization was granted for Kuvan® in 2008. Kuvan® was the first, and remains the only, medication in combination with dietary modifications in Europe designed to reduce the concentration of phenylalanine in the blood and in the brain in those patients who are responsive to Kuvan® to prevent the debilitating effects of PKU.4 Kuvan® is indicated in patients of all ages with tetrahydrobiopterin (BH4) deficiency, and in those aged 4 years and above with PKU (due phenylalanine hydroxylase enzyme deficiency) who are responsive to Kuvan. Currently, there is no licensed medication in Europe for the treatment of PKU in the 0–4 years age group. Kuvan® is marketed by Merck Serono outside the USA, Canada and Japan, by BioMarin in the USA and Canada, and under the name Biopten® by Asubio Pharma in Japan. In the USA and Europe, Kuvan® received orphan drug designation.
Disease: phenylketonuria
Details: * On October 1, 2015, Merck KGaA announced that it has reached an agreement with BioMarin Pharmaceutical to return the rights to Kuvan®, used to treat phenylketonuria (PKU), as the company focuses its healthcare business on core areas. In addition to Kuvan®, the two companies agreed that Merck will return its option to develop and commercialize Peg-Pal, an investigational drug that is also designed for the treatment of PKU, an autosomal recessive genetic disorder caused by either a defect or
a deficiency of the enzyme phenylalanine hydroxylase or its co-factor tetrahydrobiopterin. Merck had acquired the rights for Kuvan® and the option to Peg-Pal in markets outside of the U.S. and Japan from BioMarin in 2005. Since launching Kuvan® as a treatment alternative to diet alone, which constituted a paradigm shift at the time, Merck has significantly contributed to improving PKU management. More recently, Merck’s SPARK study helped pave the way for the treatment of infants with PKU below 4 years of age with Kuvan®. By returning the rights to BioMarin, Merck is confident that the product will continue to serve the best interest of the medical community and patients. A leading company in the treatment of genetic and rare diseases, BioMarin is dedicated to
improving the treatment options and to providing all resources needed to continue to make Kuvan® available, as well as to explore potential future therapies in this area, such as Peg-Pal. Merck will work closely with BioMarin during the transition to ensure
continuous access to Kuvan® for patients, physicians and health authorities.
“Returning the rights of Kuvan® and Peg-Pal to BioMarin will allow Merck to fully focus on its core businesses, as well as further align R&D investment behind key strategic areas,\" said Belén Garijo, Member of the Executive Board of Merck and CEO Healthcare. Merck remains highly committed to the patients in the field of endocrinology, and inparticular to advancing the treatment of growth hormone deficient patients with Saizen®. Over the past years, Merck has re-aligned its healthcare business with a special focus on developing novel therapies in the areas of neurology, oncology, immuno-oncology and immunology, in addition to maximizing its existing portfolio of drugs in developedcountries as well as expanding its footprint in Emerging Markets.
Financial terms: Merck will receive an upfront payment of € 340 million, equal to five times its annual sales, for Kuvan®, plus up to € 185 million in additional milestones for both products. The agreement is expected to become effective Jan. 1, 2016.
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